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New shot aims to stop liver damage in rare genetic disease

NCT ID NCT03945292

Disease control Sponsor: Arrowhead Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This Phase 2 study tested a drug called fazirsiran in 40 adults with Alpha-1 antitrypsin deficiency, a genetic condition that can cause liver disease. The drug is designed to lower levels of a harmful protein (Z-AAT) in the blood. Researchers measured safety and how much the protein dropped after 16 weeks of treatment.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
fazirsiran (also called TAK-999 or ARO-AAT), given as a shot under the skin
What this could lead to
If it works, this could lead to a treatment that lowers the toxic protein buildup in the liver, potentially slowing or stopping liver damage in people with Alpha-1 antitrypsin deficiency.
What could go wrong
This is an early (Phase 2) trial with only 40 participants, so results may not apply to everyone. The drug may not work as hoped, and side effects are still being studied.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

40 people

The number who actually took part.

Started

Aug 2019

Finished

Sep 2023

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Diagnosis of AATD * Liver biopsy at Screening indicating liver fibrosis (score less than F4); a patient with no fibrosis may participate based on a previous biopsy conducted within one year * Women of childbearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception * Willing to provide written informed consent and to comply with study requirements * Non-smoker for at least 1 year * No abnormal finding of clinical relevance at Screening Exclusion Criteria: * Clinically significant health concerns other than AATD * Previous diagnosis or diagnosis at Screening of definitive liver cirrhosis * Previous lung or liver transplant due to AATD * Regular use of alcohol within one month prior to Screening * Use of an investigational agent or device within 30 days prior to dosing or current participation in an investigational study involving therapeutic intervention * Use of illicit drugs within 1 year prior to Screening NOTE: additional inclusion/exclusion criteria may apply, per protocol

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Conditions

The condition(s) this trial relates to.

alpha 1-antitrypsin deficiency

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Alpha 1 antitrypsin deficiency

Contacts and locations

Locations

  • Research Site 1

    Charleston, South Carolina, 29425, United States

  • Research Site 10

    St Louis, Missouri, 63104, United States

  • Research Site 11

    Sacramento, California, 95817, United States

  • Research Site 12

    New York, New York, 10032, United States

  • Research Site 13

    Stanford, California, 94305, United States

  • Research Site 14

    La Jolla, California, 92037, United States

  • Research Site 15

    San Francisco, California, 94158, United States

  • Research Site 16

    Salt Lake City, Utah, 84132, United States

  • Research Site 17

    Los Angeles, California, 90095, United States

  • Research Site 19

    Houston, Texas, 77030, United States

  • Research Site 2

    Gainesville, Florida, 32610, United States

  • Research Site 20

    Barcelona, 08035, Spain

  • Research Site 22

    Pavia, 27100, Italy

  • Research Site 23

    Leiden, 2333 ZA, Netherlands

  • Research Site 24

    Funchal, 9004-514, Portugal

  • Research Site 27

    Aachen, 52074, Germany

  • Research Site 3

    Iowa City, Iowa, 52242, United States

  • Research Site 4

    Nashville, Tennessee, 37232, United States

  • Research Site 5

    Birmingham, Alabama, 35233, United States

  • Research Site 7

    Indianapolis, Indiana, 46202, United States

  • Research Site 9

    Phoenix, Arizona, 85054, United States

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